Baylor College of Medicine

A Study to Evaluate mRNA-3745 in Participants with Glycogen Storage Disease Type 1a (GSD1a)

Study coordinators who want to add a study should Submit a Clinical Trial. For more information or to remove a study, please email pa-webteam@bcm.edu. MyChart is a ...
Nasdaq

Soleno Therapeutics Receives Orphan Drug Designation from FDA for Diazoxide Choline for the Treatment of Glycogen Storage Disease Type 1a

REDWOOD CITY, Calif., June 02, 2021 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment ...
Medical News Today

What is glycogen storage disease?

Glycogen storage disease (GSD) refers to a group of rare genetic conditions that affect how the body stores and breaks down glycogen. The most common type is type I, also called von Gierke disease.
The Business Journals

Gene therapy trial shows promise in late-stage rare-disease test for Ultragenyx

To continue reading this content, please enable JavaScript in your browser settings and refresh this page. Preview this article 1 min GDS 1a afflicts only 6,000 ...
BioWorld

Ultragenyx gene therapy hits phase III goal in rare disease GSD1a

Ultragenyx Pharmaceutical Inc. anticipates a meeting later this year with the U.S. FDA to discuss a BLA filing for gene therapy DTX-401 as the first potential medical treatment for glycogen storage ...